What is Vyndaqel? | Information & FAQ| everyone.org
Last updated: 14 October 2022
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What is Vyndaqel (tafamidis)?
Vyndaqel (tafamidis) is a medicine indicated for the treatment of transthyretin amyloidosis in patients with stage 1 symptomatic polyneuropathy to delay peripheral neurologic impairment. Vyndaqel (tafamidis) is the first medication approved to stabilise the protein transthyretin (TTR) from misfolding and forming amyloids.
It is available in capsule form each containing 20mg of tafamidis.1,2
How much does Vyndaqel cost?
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What is Vyndaqel (tafamidis) used for?
Vyndaqel (tafamidis) is indicated for adults with stage 1 transthyretin familial amyloid polyneuropathy (TTR-FAP). Polyneuropathies, or peripheral neuropathies, result from damage to the peripheral nerves (nerves that go from the spinal cord to the arms, hands, legs, and feet), which often result in a sense of weakness, numbness and pain in the extremities. 3
What is transthyretin amyloidosis?
Transthyretin amyloidosis is a rare disease characterised by the presence of a defective blood protein called transthyretin (TTR). This defective protein breaks easily, which results in the deposition of a fibrous substance (amyloid fibrils) within the body. The accumulation of amyloid fibrils causes damage to body structures, like tissues and organs, and prevents their normal function. When the nerves are affected the disease is called transthyretin familial amyloid polyneuropathy (TTR-FAP). The active substance in Vyndaqel, tafamidis, is a transthyretin stabiliser. It attaches to transthyretin, the defective protein, and makes it stable. In this way, it prevents the protein from breaking up, and it may thus stop the formation of amyloid fibrils and slow down the progression of the neurological disorder. 3
Is Vyndaqel (tafamidis) approved in Canada??
Vyndaqel (tafamidis) has been approved for the treatment of transthyretin familial amyloid polyneuropathy (TTR-FAP) by the:
- EMA (EU), November 2011
- FDA (USA), May 2019
- Health Canada, January 2020
- PMDA (Japan), September 2013
- TGA (Australia), March 2020
It is also approved in Brazil, Argentina and Russia for this indication.3
What is the difference between Vyndaqel and Vyndamax?
Vyndamax contains the active drug tafamidis. Vyndaqel contains the active drug tafamidis meglumine, which is a salt form of tafamidis. Vyndamax and Vyndaqel are absorbed into the body differently, so they have different recommended dosages. They can’t be substituted on a 1:1 basis.
Who makes Vyndaqel (tafamidis)?
Vyndaqel is made by Pfizer Inc. Pfizer Inc. is an American multinational pharmaceutical corporation. Pfizer is one of the world's largest pharmaceutical companies.
References:
- Ema.europa.eu
- Pfizer.com
- Vyndaqel (tafamidis) - everyone.org.com
- Medicalnewstoday.com
